Archive for the ‘Crispr’ Category

LOS ANGELES, United States:The report titledGlobal CRISPR And CRISPR-Associated (Cas) Genes Marketis one of the most comprehensive and important additions to QY Researchs archive of market research studies. It offers detailed research and analysis of key aspects of the global CRISPR And CRISPR-Associated (Cas) Genes market. The market analysts authoring this report have provided in-depth information on leading growth drivers, restraints, challenges, trends, and opportunities to offer a complete analysis of the global CRISPR And CRISPR-Associated (Cas) Genes market. Market participants can use the analysis on market dynamics to plan effective growth strategies and prepare for future challenges beforehand. Each trend of the global CRISPR And CRISPR-Associated (Cas) Genes market is carefully analyzed and researched about by the market analysts.The market analysts and researchers have done extensive analysis of the global CRISPR And CRISPR-Associated (Cas) Genes market with the help of research methodologies such as PESTLE and Porters Five Forces analysis. They have provided accurate and reliable market data and useful recommendations with an aim to help the players gain an insight into the overall present and future market scenario. The CRISPR And CRISPR-Associated (Cas) Genes report comprises in-depth study of the potential segments including product type, application, and end user and their contribution to the overall market size.

In addition, market revenues based on region and country are provided in the CRISPR And CRISPR-Associated (Cas) Genes report. The authors of the report have also shed light on the common business tactics adopted by players. The leading players of the global CRISPR And CRISPR-Associated (Cas) Genes market and their complete profiles are included in the report. Besides that, investment opportunities, recommendations, and trends that are trending at present in the global CRISPR And CRISPR-Associated (Cas) Genes market are mapped by the report. With the help of this report, the key players of the global CRISPR And CRISPR-Associated (Cas) Genes market will be able to make sound decisions and plan their strategies accordingly to stay ahead of the curve.

Competitive landscape is a critical aspect every key player needs to be familiar with. The report throws light on the competitive scenario of the global CRISPR And CRISPR-Associated (Cas) Genes market to know the competition at both the domestic and global levels. Market experts have also offered the outline of every leading player of the global CRISPR And CRISPR-Associated (Cas) Genes market, considering the key aspects such as areas of operation, production, and product portfolio. Additionally, companies in the report are studied based on the key factors such as company size, market share, market growth, revenue, production volume, and profits.

Key Players Mentioned in the Global CRISPR And CRISPR-Associated (Cas) Genes Market Research Report:, Caribou Biosciences, Addgene, CRISPR THERAPEUTICS, Merck KGaA, Mirus Bio LLC, Editas Medicine, Takara Bio USA, Thermo Fisher Scientific, Horizon Discovery Group, Intellia Therapeutics, GE Healthcare Dharmacon

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Segmental Analysis

The report has classified the globalCRISPR And CRISPR-Associated (Cas) Genesindustry into segments including product type and application. Every segment is evaluated based on growth rate and share. Besides, the analysts have studied the potential regions that may prove rewarding for theCRISPR And CRISPR-Associated (Cas) Genes manufacturers in the coming years. The regional analysis includes reliable predictions on value and volume, thereby helping market players to gain deep insights into the overallCRISPR And CRISPR-Associated (Cas) Genesindustry.

GlobalCRISPR And CRISPR-Associated (Cas) Genes Market Segment By Type:

, the CRISPR And CRISPR-Associated (Cas) Genes market is segmented into, Genome Editing, Genetic engineering, gRNA Database/Gene Librar, CRISPR Plasmid, Human Stem Cells, Genetically Modified Organisms/Crops, Cell Line Engineering

GlobalCRISPR And CRISPR-Associated (Cas) Genes Market Segment By Application:

Biotechnology Companies, Pharmaceutical Companies, Academic Institutes, Research and Development Institutes

The CRISPR And CRISPR-Associated (Cas) Genes Market report has been segregated based on distinct categories, such as product type, application, end user, and region. Each and every segment is evaluated on the basis of CAGR, share, and growth potential. In the regional analysis, the report highlights the prospective region, which is estimated to generate opportunities in the global CRISPR And CRISPR-Associated (Cas) Genes market in the forthcoming years. This segmental analysis will surely turn out to be a useful tool for the readers, stakeholders, and market participants to get a complete picture of the global CRISPR And CRISPR-Associated (Cas) Genes market and its potential to grow in the years to come.

Key questions answered in the report:

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Table of Contents:

Table of Contents 1 Report Overview1.1 Research Scope1.2 Top CRISPR And CRISPR-Associated (Cas) Genes Manufacturers Covered: Ranking by Revenue1.3 Market Segment by Type

1.3.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Type: 2015 VS 2020 VS 2026 (US$ Million)

1.3.2 Genome Editing

1.3.3 Genetic engineering

1.3.4 gRNA Database/Gene Librar

1.3.5 CRISPR Plasmid

1.3.6 Human Stem Cells

1.3.7 Genetically Modified Organisms/Crops

1.3.8 Cell Line Engineering1.4 Market Segment by Application

1.4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Consumption by Application: 2015 VS 2020 VS 2026

1.4.2 Biotechnology Companies

1.4.3 Pharmaceutical Companies

1.4.4 Academic Institutes

1.4.5 Research and Development Institutes1.5 Study Objectives1.6 Years Considered 2 Global Market Perspective2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2015-2026)

2.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue (2015-2026)

2.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales (2015-2026)2.2 CRISPR And CRISPR-Associated (Cas) Genes Market Size across Key Geographies Worldwide: 2015 VS 2020 VS 2026

2.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Regions (2015-2020)

2.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Regions (2015-2020)2.3 Global Top CRISPR And CRISPR-Associated (Cas) Genes Regions (Countries) Ranking by Market Size2.4 CRISPR And CRISPR-Associated (Cas) Genes Industry Trends

2.4.1 CRISPR And CRISPR-Associated (Cas) Genes Market Top Trends

2.4.2 Market Drivers

2.4.3 CRISPR And CRISPR-Associated (Cas) Genes Market Challenges 2.4.4 Porters Five Forces Analysis

2.4.5 Primary Interviews with Key CRISPR And CRISPR-Associated (Cas) Genes Players: Views for Future 3 Competitive Landscape by Manufacturers3.1 Global Top CRISPR And CRISPR-Associated (Cas) Genes Manufacturers by Sales (2015-2020)

3.1.1 Global CRISPR And CRISPR-Associated (Cas) Genes Sales by Manufacturers (2015-2020)

3.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Manufacturers (2015-2020)

3.1.3 Global 5 and 10 Largest Manufacturers by CRISPR And CRISPR-Associated (Cas) Genes Sales in 20193.2 Global Top Manufacturers CRISPR And CRISPR-Associated (Cas) Genes by Revenue

3.2.1 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue by Manufacturers (2015-2020)

3.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Share by Manufacturers (2015-2020)

3.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Market Concentration Ratio (CR5 and HHI)3.3 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in CRISPR And CRISPR-Associated (Cas) Genes as of 2019)3.4 Global CRISPR And CRISPR-Associated (Cas) Genes Average Selling Price (ASP) by Manufacturers3.5 Key Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Plants/Factories Distribution and Area Served3.6 Date of Key Manufacturers Enter into CRISPR And CRISPR-Associated (Cas) Genes Market3.7 Key Manufacturers CRISPR And CRISPR-Associated (Cas) Genes Product Offered 3.8 Mergers & Acquisitions, Expansion Plans 4 Market Size by Type4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Historic Market Review by Type (2015-2020)

4.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Type (2015-2020)

4.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Type (2015-2020)

4.1.4 CRISPR And CRISPR-Associated (Cas) Genes Price by Type (2015-2020)4.1 Global CRISPR And CRISPR-Associated (Cas) Genes Market Estimates and Forecasts by Type (2021-2026)

4.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Type (2021-2026)

4.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Type (2021-2026)

4.2.4 CRISPR And CRISPR-Associated (Cas) Genes Price Forecast by Type (2021-2026) 5 Global CRISPR And CRISPR-Associated (Cas) Genes Market Size by Application5.1 Global CRISPR And CRISPR-Associated (Cas) Genes Historic Market Review by Application (2015-2020)

5.1.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Market Share by Application (2015-2020)

5.1.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Market Share by Application (2015-2020)

5.1.4 CRISPR And CRISPR-Associated (Cas) Genes Price by Application (2015-2020)5.2 Global CRISPR And CRISPR-Associated (Cas) Genes Market Estimates and Forecasts by Application (2021-2026)

5.2.2 Global CRISPR And CRISPR-Associated (Cas) Genes Sales Forecast by Application (2021-2026)

5.2.3 Global CRISPR And CRISPR-Associated (Cas) Genes Revenue Forecast by Application (2021-2026)

5.2.4 CRISPR And CRISPR-Associated (Cas) Genes Price Forecast by Application (2021-2026) 6 North America6.1 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company6.2 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type6.3 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application6.4 North America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

6.4.1 North America CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

6.4.2 North America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

6.4.3 U.S.

6.4.4 Canada 7 Europe7.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company7.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type7.3 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application7.4 Europe CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

7.4.1 Europe CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

7.4.2 Europe CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

7.4.3 Germany

7.4.4 France

7.4.5 U.K.

7.4.6 Italy

7.4.7 Russia 8 Asia Pacific8.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company8.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type8.3 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application8.4 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Regions

8.4.1 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Sales by Regions

8.4.2 Asia Pacific CRISPR And CRISPR-Associated (Cas) Genes Revenue by Regions

8.4.3 China

8.4.4 Japan

8.4.5 South Korea

8.4.6 India

8.4.7 Australia

8.4.8 Taiwan

8.4.9 Indonesia

8.4.10 Thailand

8.4.11 Malaysia

8.4.12 Philippines

8.4.13 Vietnam 9 Latin America9.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Company9.2 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type9.3 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application9.4 Latin America CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

9.4.1 Latin America CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

9.4.2 Latin America CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

9.4.3 Mexico

9.4.4 Brazil

9.4.5 Argentina 10 Middle East and Africa10.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Type10.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Application10.3 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Breakdown Data by Countries

10.3.1 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Sales by Countries

10.3.2 Middle East and Africa CRISPR And CRISPR-Associated (Cas) Genes Revenue by Countries

10.3.3 Turkey

10.3.4 Saudi Arabia

10.3.5 U.A.E 11 Company Profiles11.1 Caribou Biosciences

11.1.1 Caribou Biosciences Corporation Information

11.1.2 Caribou Biosciences Business Overview and Total Revenue (2019 VS 2018)

11.1.3 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)

11.1.4 Caribou Biosciences CRISPR And CRISPR-Associated (Cas) Genes Products and Services

11.1.5 Caribou Biosciences SWOT Analysis

11.1.6 Caribou Biosciences Recent Developments11.2 Addgene

11.2.1 Addgene Corporation Information

11.2.2 Addgene Business Overview and Total Revenue (2019 VS 2018)

11.2.3 Addgene CRISPR And CRISPR-Associated (Cas) Genes Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)

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CRISPR And CRISPR-Associated (Cas) Genes Market Break Down by Top Companies, Countries, Applications, Challenges, Opportunities and Forecast by...

CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) have announced that the European Medicines Agency has granted their experimental, autologous, ex vivo CRISPR/Cas9 gene-edited therapy, CTX001 Priority Medicines designation. The therapy has been granted PRIME designation for severe sickle cell disease treatment.

The PRIME designation is a regulatory mechanism that offers early and proactive support to promising medicines developers to enhance development plans. Also, it accelerates the evaluation of medicines under development to reach patients as soon as possible. PRIMEs objective is to ensure patients benefit fasters from new proprietary therapies that have shown the potential of addressing a significant unmet medical need. The companies received the PRIME designation based on data from their on-going phase 1/2 study of CTX001 in treating severe sickle cell disease patients.

CTX001 is currently being investigated to treat patients with severe SCD or transfusion-dependent beta-thalassemia, whereby hematopoietic stem cells are purposed to produce high fetal hemoglobin levels in red cells. HbF is an oxygen-carrying hemoglobin form present naturally at birth, which later switches to adult hemoglobin form. HbF elevation by CTX001 can potentially alleviate transfusion requirements for TDT patients and minimize painful and devastating sickle crises in patients with SCD.

So far, CTX001 has received Regenerati9ve Medicine Advanced Therapy (RMAT), Orphan Drug Designation, and Fast Track designation from the FDA. Also, CTX001 has Orphan Drug Designation from the European Commission for SCD and TDT.

CRISPR Therapeutics and Vertex are developing CTX001 under a co-development and co-commercialization agreement. So far, CTX001 is the most progressive gene editing therapy under development for SCD and TDT. The companies entered a strategic collaboration agreement in 2015 intending to use CRISPR/Cas9 to develop and discover novel therapies for the treatment of underlying genetic causes of diseases. CTX001 is the first joint treatment to emerge from the collaboration, and all development costs and profits will be shared.

Read this article:
CRISPR Therapeutics (NASDAQ:CRSP) and Vertex (NASDAQ:VRTX) Receive Priority Medicines (PRIME) Designation From EMA For CTX001 In SCD - BP Journal

Emmanuelle Charpentier and Jennifer Doudna have been given the 2020 Nobel Prize in Chemistry for their discovery and development of CRISPR-Cas9 genome editing.

The Royal Swedish Academy of Sciences has decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens, Germany, and Jennifer Doudna of the University of California, Berkeley, US. The Nobel Prize is for the development of CRISPR-Cas9, a method for genome editing.

According to the Royal Swedish Academy of Sciences, Emmanuelle Charpentier and Jennifer Doudna discovered the CRISPR-Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may aid in curing inherited diseases.

Also, using the CRISPR-Cas9 genetic scissors, it is now possible to change DNA over the course of a few weeks. This used to be time-consuming, difficult and sometimes impossible work.

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments, said Claes Gustafsson, chair of the Nobel Committee for Chemistry.

The discovery of CRISPR-Cas9 was made during Emmanuelle Charpentiers studies of Streptococcus pyogenes, one of the bacteria that cause the most harm to humanity. She discovered a previously unknown molecule, tracrRNA. Her work showed that tracrRNA is part of bacterias ancient immune system, CRISPR-Cas, that disarms viruses by cleaving their DNA.Charpentier published her discovery in 2011. The same year, she initiated a collaboration with Jennifer Doudna, an experienced biochemist with vast knowledge of RNA. Together, they succeeded in recreating the bacterias genetic scissors in a test tube and simplifying the scissors molecular components so they were easier to use.

In another experiment, they then reprogrammed the genetic scissors. In their natural form, the scissors recognise DNA from viruses, but Charpentier and Doudna proved that they could be controlled so that they can cut any DNA molecule at a predetermined site.

The academy highlights that since Charpentier and Doudna discovered the CRISPR-Cas9 genetic scissors in 2012 their use has exploded. This tool has contributed to many important discoveries in basic research and clinical trials of new cancer therapies are underway.

Continued here:
Nobel Prize in Chemistry awarded to discoverers of CRISPR-Cas9 - Drug Target Review

Graphic Source: CRISPR Therapeutics, Inc.

CRISPR Therapeutics (NASDAQ:CRSP) is a gene-editing company focused on the development and versatile application of CRISPR/Cas9 therapeutics, a special brand of therapeutics used for precision genome editing by applying a viral defense mechanism from bacteria to regulate, disrupt, or correct genes related to key diseases. CRSP is currently targeting disease areas, including hemoglobinopathies, oncology, and regenerative medicines.

Founded in 2013 in Switzerland, CRSP has since grown to over 304 employees producing relatively inconsistent revenues ranging from $3M in 2018 to $290M in 2019 with expectations for 2020 at $6.7M. Their lead candidate is CTX001, an investigational autologous gene-edited hematopoietic stem cell therapy developed in partnership with Vertex Pharmaceuticals (NASDAQ:VRTX) for treating transfusion-dependent beta-thalassemia ("TDT") and severe sickle cell disease ("SCD").

Products: CRSP's pipeline consists of 9 therapeutics: 4 in the clinical phase and 5 in the research phase. Of the 4 clinical phase therapeutics, the first targets TDT and SCD (mentioned above: CTX001), while the 3 others fall into immuno-oncology covering: CD19+ malignancies (Product: CTX110), multiple myeloma (CTX120) and solid tumors and hematologic malignancies (CTX130). All immuno-oncology therapeutics are allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapies wholly owned by CRISPR Therapeutics with data updates typically every 6 months.

Customers/market: For CRSP's clinical phase pipeline, the total estimated 2022 global market potential is $220B with an average market size for each disease of $36.7B growing at an average 15.2% CAGR (median market: $13.3B | CAGR 10.9%). The largest market is Solid Tumors, at a 2022 estimated size of $145B (8.1% CAGR), and the highest CAGR market CAR T/CD19+ market at a 34.5% CAGR. For CTX001, the lead candidate, the target market can be broken down into the TDT market at very roughly $1.8B with a 10.8% CAGR and the SCD market at $4.1B with an 11% CAGR by 2022.

Management: Many are now familiar with co-founder Dr. Emmanuelle Charpentier, who in 2020 under much-debated circumstances co-received the Nobel Prize in Chemistry for her work with developing the CRISPR/Cas9 genetic scissors, the foundation of CRSP's therapeutics today. In addition to her role now as Scientific Advisory Board Member, CRSP has a variety of other accomplished leaders.

CEO: Dr. Samarth Kulkarni has served as CEO (covering long-term strategy) since December of 2017 when he was promoted from President and Chief Business Officer. Before CRSP, he was a Partner at McKinsey & Company (MCK) co-leading the biotech practice. His specialties are in strategy and operations, and he has a Ph.D. in Bioengineering and Nanotechnology.

Share Price Change under his leadership (Dec. 2017 - Present): 499% | CAGR: ca 71%.

President/Chairman: Dr. Rodger Novak, a co-founder with Charpentier and Shaun Foy in 2013, has served as CEO until 2017 and since, as President (day-to-day operations) and Chairman. Rodger is an experienced biotech/pharma executive having served in leadership positions (primarily covering infectious diseases and related) at Sanofi and Novartis. He co-also founded Nabriva Therapeutics (NASDAQ:NBRV). His specialty is in translating scientific technologies into pharmaceutical products. Before all else, he was a professor of Microbiology at the Vienna BioCenter (Austria).

Other management updates:

Strategy: In terms of strategy, CRSP stated they intend to use their scientific expertise, together with their unique platform to bring about a new class of highly active and potentially curative therapies for specialty patients to which biopharmaceutical approaches have limited exposure. CRSP has been investing heavily in its long-term platform.

Additionally, CRSP seems to be taking the lead in most of its partnerships, particularly the ViaCyte partnership evidenced by the structure of their collaboration agreements and payments due to ViaCyte. Investors also saw last year CRSP buying Bayer's (OTCPK:BAYZF) control of Casebia and expanding their internal clinical pipeline with internal funds (hence the cash stockpile). CRSP is acting as the all-or-nothing winner. It is a unique approach that expresses internal confidence in their technology and financial capabilities.

Financial position: CRSP received new upfront payments from Vertex in 2019 boosting revenues to $290M, which are not expected to be repeat. The estimated decline in 2020 brings revenue to $6.7M (-98%). 2019 was the only year in the company's history to achieve net income ($67M), whereas the 3-year average net loss is -$56M. For 1H 2020, net losses are already -$149M. CRSP operates with a strong cash cushion of $945M at 1H 2020, enough to cover the -$42M 3-year average CFO+CAPEX expenditures for 22+ years. Total debt as of 1H 2020 is a manageable $50M ($40M in capital leases). Accounts payable are a small $13M.

Investment thesis: Although most of CRSP's products are years away from revenue-generating outside of milestone payments, the constant updates from clinical trials offer a compelling progress report for the long position. Investors must be willing to pay for the premium that exists currently, but with CRISPR being in the public spotlight, investor interest may increase. The real question remains if they are truly the most advanced CRISPR position, though clearly not discounted. Operational strategy is a key selling point with the new McKinsey-inherited leadership, but science is still the core of any biotech investment. Below will be an expounded analysis of what their therapeutics are, but it seems to be that CRSP has a compelling niche for the long-term investor, given their 7+ years of experience with this new biotechnology. Therefore, with the Vertex partnership, enough cash to keep steady progress, and stable operational-based leadership, CRSP is a "buy".

CRSP's pipeline consists of 9 therapeutics: 4 clinical phases and 5 in the research phase. CRSP is supported by 1 strong partnership and 1 weaker partnership including:

Vertex partnership (est. 2015) for clinical analysis of TDT and SCD with a research phase target of cystic fibrosis ("CF") is based around co-developing CTX001 (since Dec. 2017). The partnership did expand in June 2019 for Duchenne muscular dystrophy ("DMD") and myotonic dystrophy type 1, which adds further upside potential for CRSP.

In 2015, CRSP received a $75M upfront payment and in 2017 received $7M and, thereafter, a low-seven-digit milestone payment for second patient dosing related to TDT and SCD. Looking forward, CRSP is eligible for up to $420M for further milestones and product-sales royalties.

In 2019, after the new collaboration agreement was established for DMD and myotonic dystrophy type 1 ("DM1"), CRSP has received an upfront payment of $175M with eligible milestone payments up to $825M. Tiered royalties on product sales are also available. The DMD program makes Vertex responsible for R&D, manufacturing, and commercialization. For DM1, CRSP will cover RNA research with Vertex responsible for all other costs. After DM1 IND filing, CRSP retains the option to co-develop/co-commercialize all DM1 products but must forgo milestone payments/royalties and cover 50% of R&D costs incurred by Vertex. Similar amendments were made to the 2015 agreement. In Oct. 2019, Vertex accepted the right to exclusively license the three remaining options granted under their 2015 agreement, resulting in CRSP receiving a $30M 4Q 2019 payment. CRSP also received a milestone $25M payment in 2Q 2020. At 1H 2020, CRSP had $11.8M of non-current deferred revenue related to Vertex.

ViaCyte partnership (est. Sep 2018) for diabetes through gene-edited allogeneic stem cell therapies. A key aspect of this partnership is ViaCytes stem cell capabilities and CRSP's gene-editing capabilities to enable beta-cell replacement without the need for immune system suppression. After successful completion of the studies proving verification of developing the immune-evasive stem cell line, CRSP and ViaCyte will jointly be responsible for further development and commercialization, globally. The partnership entitled ViaCyte to $16.2M in payments for participating which CRSP recognized as $15M in R&D expense and $1.2M in other expenses. The expected partnership term is in force for 5.5+ years and obligates both parties to jointly develop the research plan with each party responsible for their research costs.

Bayer partnership (JV est. 2015) was terminated in 4Q 2019 which included Casebia Therapeutics and focused on treating genetic causes related to bleeding disorders and autoimmune diseases, amongst others. CRSP retained full-ownership of Casebia and Bayer retained the right to co-develop two therapeutics related to autoimmune disorders, eye disorders, and hemophilia A disorders with exclusive licenses; termed the "2019 Option Agreements".

The clinical phase therapeutics of CRSP will be outlined below.

Graphic Source: CRISPR Therapeutics Investor Presentation (Sep 2020)

Therapeutic 1: CTX001 is CRSP's lead candidate targeting TDT and severe SCD through an investigational autologous gene-edited hematopoietic stem cell therapy. It is being co-developed by Vertex Pharmaceuticals.

TDT: CTX001 is currently in a Phase 1/2 open-label clinical trial (CLIMB THAL-111) for transfusion-dependent -thalassemia. The study aims to assess safety and efficacy for single dosages of CTX001 in a 12-35-year-old population with TDT. In 4Q 2019, Vertex & CRSP expanded the TDT patient population to include beta 0/beta 0 subtypes with the first two severe patients indicating successful dosing and engraftment. The study is designed for up 45 patients and aims to follow them for a duration of two years post-infusion with 6-month investor updates. CTX001 has received the Regenerative Medicine Advanced Therapy ("RMAT"), fast-track, and orphan drug designations (+European Commission) by the FDA for treating TDT. Preliminary clinical data was released in 4Q 2019, and in June 2020, their 15 months of follow-up data were also released in the ongoing study.

SCD: CTX001 is also currently in a Phase 1/2 open-label clinical trial (CLIMB SCD-121) for severe sickle cell disease testing safety and efficacy for single dosages of CTX001 in an older patient population than TDT (18-35). Similar to CLIMB THAL-111, the first two patients were treated sequentially then expanded for up to 45 concurrent patients for a 2-year following. CTX001 for SCD has also received the same designations as for TDT with the same data publication dates but in June releasing only 9 months of follow-up data.

Competition Notes:

Therapeutic 2: CTX110 is CRSP's lead candidate amongst their wholly-owned CAR-T therapies, which is a gene-edited allogeneic CAR-T therapy targeting CD19 in CD19+ malignancies cases. It's currently in a Phase 1 study focused on safety and efficacy ("S&E") in the treatment of relapsed/refractory B-cell malignancies. The study is designed for up to 131 patients on a multi-dose level.

Therapeutic 3: CTX120 is another gene-edited allogeneic CAR-T therapy but targeting B-cell maturation antigen. CTX120 is in a Phase 1 S&E study for treating relapsed/refractory("R&R") multiple myeloma. It is also a multi-center open-label trial but designed for up to 88 patients also on a multi-dose level investigation.

Therapeutic 4: CTX130 is CRSP's other CAR-T therapy with a target of CD70, the antigen expressed on solid-tumors and hematologic malignancies (study's target). The treatment is developed around solid-tumors (e.g. renal cell carcinoma) and T/B-cell hematologic malignancies. CRSP is currently running two independent Phase 1 S&E studies for CTX130 treating R&R renal cell carcinoma and other types of lymphoma. The first Phase 1 study, focused on R&R renal cell carcinoma, is a multi-center open-label investigation with an enrollment of up to 95 patients on a multi-dose level, and the second study for various lymphomas is designed for up to 46 patients.

For further analysis of the science and clinical trial updates, see clinical.gov studies linked above, the September 2020 Investor presentation, or the Chardan Conference Call (Sep 2020).

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Revenue/cash flow: Financially, CRSP is far from stable on any operating metric (other than cash resources), but survived COVID with 80-90% productivity. Revenue is earned from collaboration agreements and their associated milestones. This swayed investors in 2019 when $289M (99.6% of 2019 Revenue) was recognized from the Vertex partnership expansion. This comprised revenue from the DMD and DM1 licenses worth $202M and from Vertex exercising their "Collaboration Target Options" worth $76.7M and a $6.7M payment for Vertex waving their fourth exclusive license. Neither CRSP nor the author expects this to continue in the next 1-2 years on any metric unless new partnerships are formed and upfront payments are made.

CRSP does have $12.6M in unearned revenue accounted for, but only 1M as current. Analysts do expect another $6.5M in revenue 2H 2020, but that only brings the tangible revenue benchmark to $6.7M for the year; however, the focus is on the viability of the therapeutics and not revenue benchmarks. It seems investors in CRSP think the same. New and old investors should focus on the results of clinical trials for the foundation of any investment thesis as accessing the Vertex milestone payments worth up $1.25B is directly the result of the Phase 1/2 CTX001 results and the Pre-IND research phase targeting DMD, DM1, and CF.

Balance sheet composition:

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Regarding the balance sheet, what stands out for investors is the high cash balance accumulated ($945M), particularly from Vertex, which is sufficient to finance the existing pipeline of 4-core products (3 internal, 1 external) and a few promising research phase therapeutics. The low-unearned revenue ($13M) enlightens investors towards the short term, but with little debt ($50M) and capital leases making up the majority portion ($44M), there isn't a heavy draw on cash that shouldn't produce an advancement towards reaching clinical phases. What is worrisome though is the desire for only 2 external partners (1-lead and 1-sub) which may be attributed to the McKinsey style leadership that in the author's opinion does not foster the right developmental environment and deviates from the biotech norm. This may be a benefit in some investors' eyes as the rewards will be substantial in 3-7 years, but it is a long-term position an investor must make.

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Firstly, any valuation on CRSP at this point is highly speculative, given the inconsistent milestone marks being met and their highly divergent payments with relatively new technology (though bluebird and Novartis are setting precedents). A revenue basis seems the most closely tied to reality and by using 11-14 analyst forecasts, an approximate valuation can be made as above with base-case (+2% upside) being far too conservative due to the reactions investors take upon clinical trial announcements and certainly not enough to lure any biotech investor.

An upside of 311% in an optimistic scenario seems more than compensatory to the risk, given the large cash cushion. However, a premium of 894x Sales is remarkable, but outlandishly inaccurate due to the fluctuations of the revenue position which does not compensate for the high potential of gaining market share in the $220B market their combined therapeutics target. In summary, the author will say that an upside potential exists but is uncertain beyond 30% in the short term (1-2 years).

Data by YCharts

Upcoming Catalysts (1-12 months):

In summary, CRSP is in a very unique position with such a large $945M cash cushion and a promising advancement on therapeutic development with cutting-edge technology and 7+years of experience in it, far greater than most enterprising biotechs looking into genome-editing. The partnership with Vertex, a leader in its sphere, does provide substantiation to the technology CRSP is applying, but with only two partnerships, it seems CRSP is either too early to the game or is not moving fast enough. CRSP does face competition, such as bluebird's TDT gene-therapy; however, what CRSP is building should surpass competition if it can swiftly make it to market compensating investors that must withstand early-clinical phase results risk. CRSP has stated their platforms are worthy of reaching a $100B company status, but the author adds that investors must be patient. There are few companies that the author truly feels uncertain regarding valuing, and CRSP is one of them. Any realistic valuation would greatly underestimate the potential of CRSP, but by averaging the downside and the upside potential, a reasonable price target can be surmised from analyst expectations. Therefore, the author projects CRISPR Therapeutics as a "buy" for the long-term investor with an uncertain 2-year stock price target at $204.63 (+113% upside).

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Crispr Therapeutics: Waiting On Early Data In 2H 2020, But The Clinical Pipeline Shows Promise (113% Upside) - Seeking Alpha

Researchers in the US led by the 2020 Nobel laureate for chemistry Jennifer Doudna have used a CRISPR gene-editing technology to develop a rapid, portable, accurate, and low-cost mobile-based test that can detect the pandemic coronavirus (Sars-CoV2) in five minutes.

This new CRISPR diagnostic method doesnt amplify coronavirus RNA but uses multiple guide RNAs that work in tandem to increase the sensitivity of the test, said the research team in the yet to be peer-reviewed study published in the pre-print server medRxiv. The test does not require expensive lab equipment, and can be deployed for rapid point-of-care testing at doctors offices, schools, and office buildings.

The diagnostic gold standard for coronavirus disease diagnosis is the quantitative reverse transcription-polymerase chain reaction (RT-qPCR) test, which takes five to six hours to produce result. CRISPR-based diagnostics that utilises RNA and DNA-targeting enzymes can augment gold-standard PCR-based testing if they can be made rapid, portable and accurate, said Doudnas team.

The assay achieved ~100 copies/L sensitivity in under 30 minutes and accurately detected a set of positive clinical samples in under 5 minutes. We combined crRNAs targeting SARS-CoV-2 RNA to improve sensitivity and specificity, and we directly quantified viral load using enzyme kinetics. Combined with mobile phone-based quantification, this assay can provide rapid, low-cost, point-of-care screening to aid in the control of SARS-CoV-2, write researchers.

The test also quantifies the amount of virus in a sample, with the strength of the fluorescent signal proportional to the amount of virus in a sample. This cannot be done in standard Covid-19 tests that amplify the virus genetic material to detect it. Detecting a patients viral load can guide treatment decisions. The test needs validation before it is commercially available.

CRISPR diagnosis work by identifying a sequence of RNAabout 20 RNA bases longthat is unique to SARS-CoV-2. They do so by creating a guide RNA that is complementary to the target RNA sequence so it binds to it in solution. The binding turns on the CRISPR tools Cas13 scissors enzyme that cuts single-stranded RNA to release a separately introduced fluorescent particle in the test solution. These fluorescent particles light up to when hit with laser light, signaling the presence of the virus.

CRISPR diagnostics is already being used for Sars-CoV-2 detection, but the new test is the fastest CRISPR-based diagnostic yet.

I think this is an interesting new approach that is faster because it doesnt have an amplification step. But, because it doesnt have the amplification step, it cannot easily detect low viral loads unlike qRT-PCR or FELUDA. The five minutes result is only when starting from RNA with high amount of virus. For usual samples it will be more than 30 minutes. The device requirement is not zero but low - a constant temperature holder and a laser illumination optical box. I think CRISPR based tests will see a lot of innovation and it is a good sign for the fight against COVID-19, said Dr Anurag Agrawal, director, Council of Scientific & Industrial Research-Institute of Genomics and Integrative Biology (CSIR-IGIB), New Delhi.

Scientist from CSIR-IGIB have also developed a precise and cost-effective strip test named after a popular Satyajit Ray detective FELUDA to detect Covid-19 in one hour, starting from RNA to giving a visual readout on the strip.

FELUDA, which is an acronym for FNCAS9 Editor-Linked Uniform Detection Assay, uses CRISPR gene-editing technology to identify and target the genetic material of Sars-CoV2, the virus that causes Covid-19. It has been developed by CSIR-IGIB senior scientists Dr Debojyoti Chakraborty and Dr Souvik Maiti.

FELUDA tests work by combining CRISPR biology and paper strip chemistry. A Cas9 protein, a component of the CRISPR system, is barcoded to interact specifically with the Sars-CoV2 sequence in the patients genetic material.

The complex of Cas9 with Sars-CoV2 is then applied to a paper strip, where using two lines (one control, one test) make it possible to determine if the test sample was infected with Covid-19.

Going forward, CRISPR-based tests have the potential for modification to detect the next emerging virus and rapidly scale up testing, if needed.

The rest is here:
Covid detection with CRISPR, phones in offing - Hindustan Times

With the vision of linking computers up to the LIVE signals of biology, Cardea now adds an advisory team of Key Opinion Leaders covering all the cross disciplinary efforts being done.

SAN DIEGO (PRWEB) October 13, 2020

Cardea Bio Inc., who is using graphene-based Biology-gated Transistors (Cardean Transistors) to directly link the live signals that run biology up to electronics and computers, today announced the Cardea Innovation Council. The Council will serve as an advisory body to guide and help the Cardea team of talents to continue the breakthroughs being made via Cardea's core technology, Cardean Transistors. The council members will also participate in the Company's Innovation Partnership Program on relevant projects. The Council consists of a body of Key Opinion Leaders and experts from diverse science and technical fields and will bring a depth of knowledge to aid Cardea in building the most complete Tech+Bio Communication Chipsets and Infrastructure available for current and future generations.

Cardea is pleased to welcome the Council Members to its team:

Professor Susan Wessler

Distinguished Professor of Genetics and the Neil and Rochelle Campbell Chair for Innovation in Science Education at the University of California Riverside. In 2011 she was elected Home Secretary of the U.S. National Academy of Sciences (NAS), the first woman to hold this position in its 150-year history. She is a plant molecular geneticist known for her contributions to the field of transposon biology and plant genome evolution.

Professor Virginijus Siksnys

Among the very first to discover and characterize the CRISPR-Cas9 complex and recognize its editing potential for "DNA surgery" in many life science applications. His pioneer work was recognized with several international awards including the Kavli Prize. Since CRISPR-Chip is an important chipset type for Cardea, his expertise regarding CRISPR is important in improving fast and precise (amplification-free) DNA and RNA detection.

Dr. Kurt Petersen

Founder and CTO of numerous MEMS (Micro-ElectroMechanical System) companies, including NovaSensor, Verreon and molecular testing company Cepheid. Kurt was recently awarded the IEEE Medal of Honor for his contribution to the field of MEMS. Cardean Transistors are very similar to MEMS in many regards and Kurt's experience with MEMS as it applies to biotechnology will elevate Cardea's chip designs, scalability and capabilities.

Dr. Phil Cotter

Fellow of the American College of Medical Genetics and Genomics as well as founder and Principal of ResearchDx. Dr. Cotter has been a leader in developing sequencing as a clinical diagnostics tool as the Director of Illumina Clinical Services Laboratory, and through ResearchDx, a leader in development of companion diagnostics based on DNA, RNA and protein biomarkers.

Dr. Lauge Farnaes

As Head of Medical Affairs at IDbyDNA, and Medical Doctor & Researcher formally at Rady Children's Genomic Institute with expertise in genetic disorders, infectious disease detection, and nucleic acid-based diagnostics, Dr. Farnaes has among other things helped pioneer the use of Rapid Whole Genome Sequencing to diagnose rare genetic disorders in children.

Dr. Elia Stupka

A visionary Key Opinion Leader in bringing the most advanced forms of big data and analytics to healthcare. Dr. Stupka's work has contributed to the understanding of the human genome, transcriptome, and the development of gene-therapeutics. His understanding of data and its role in enhancing the value of new technology will help steer Cardea's data management capabilities and add value to every Innovation Partner application.

Dr. Paul Grint

Chairman of the Innovation Council and Chairman of the Board of Directors of Cardea. Dr. Grint has served on the Boards of Illumina, AmpliPhi Biosciences, and as the CEO of several companies. Dr. Grint's ability to see the early potential in technology served Illumina well when Next Generation Sequencing was in its infancy as it will Cardea with its Biology-gated Transistors.

"Cardea's core technology is really the convergence of many highly complex technical fields such as life science, data analytics, and semiconductor technology." says Dr. Grint. "In order for Cardea to be successful in its mission to elevate the world's ability to gain new insight into biology, we need the best from every field. The Innovation Council will elevate Cardea's capabilities across all of these fields."

The news of Cardea's Innovation Council comes only weeks after the company announced the first close of their A2 round and first commercial "Powered by Cardea" product launch. This is a major move for Cardea on its mission to continue developing even more products together with their Innovation Partners. To learn more about the Council, visit Cardea's website.

About Cardea Bio

Cardea is linking biology directly up to computers for the very first time by building a Tech+Bio Infrastructure and offering chipsets based on proprietary Biology-gated Transistors, or Cardean Transistors. These transistors leverage graphene, a nanomaterial that in contrast to the common semiconductor material silicon, is biocompatible and a near perfect conductor due to only being one atom thick. It that way replaces optical static observations with interactive live-streams of multi-omics signal analysis, representing a new life science observation paradigm where multi-omics data-streams will be the new norm instead of most of the current standard technologies that are single-omics frozen-in-time datasets. Together with their Innovation Partners, Cardea can link biology directly to compute power and convert real-time biological signals to digital information, allowing for immediate biological insight and a new generation of applications Linking up to Life.

Contacts

Partnership inquiries

Rob Lozuk

Chief Business Officer

PublicRelations@Cardeabio.com

Media inquiries

Amanda Zimmer

Marketing Manager

marketing@cardeabio.com

For the original version on PRWeb visit: https://www.prweb.com/releases/crispr_pioneer_home_secretary_of_the_u_s_national_academy_of_sciences_ieee_medal_of_honor_recipient_and_other_experts_joins_cardea_bios_innovation_council/prweb17465996.htm

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CRISPR Pioneer, Home Secretary of the U.S. National Academy of Sciences, IEEE Medal of Honor Recipient, and other experts joins Cardea Bio's...

Scribe Therapeutics, a biotech firm focused on developing next-generation gene-editing technology, has raised $20 million in its first major round of financing, backed by Andreessen Horowitz. The firm separately unveiled a deal with Biogen to develop CRISPR-based treatments for amyotrophic lateral sclerosis (ALS).

Scribe was cofounded in 2018 by several University of California, Berkeley, scientists, including gene-editing pioneer Jennifer Doudna and protein engineer Benjamin Oakes, who at the time was an entrepreneurial fellow at the Innovative Genomics Institute, where Doudna is president. Their goal was to engineer a newly discovered class of Cas proteins to make them behave better as therapies than the original CRISPR-Cas9 gene-editing system.

The original system was found in bacteria, which use it to recognize and chop up DNA from invading pathogens. Scientists, including Doudna, quickly realized the system could be co-opted to make precise cuts to human DNA. The tool set off a race among companies trying to use it to address the genetic mutations underlying many diseases.

But even with its promise, the CRISPR-Cas9 system comes with evolutionary baggage, Oakes, who is now CEO of Scribe, says. Those systems arent designed to work within the context of the human cell or even the human genome, he says, complicating efforts to turn the technology into drugs.

Read more from the original source:
Scribe Therapeutics launches to explore next-generation CRISPR technology - Chemical & Engineering News

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CRISPR Industry Size 2019, Market Opportunities, Share Analysis up to 2026 - The Research Process

Several years ago, a brand new method of genetic engineering called CRISPR was invented, and it was based on discoveries made about the rudimentary "immune system" possessed by bacteria. Essentially, bacteria have a way of "remembering" which viruses had infected them previously, and they possess a molecular system that destroys viral DNA that matches that of a prior infection.

The molecular system consists of a DNA-cutting protein called Cas9. (See infographic from Business Insider below.) When equipped with a special guide RNA, Cas9 can be used to cut specific DNA sequences, for instance, a mutated gene that is causing a health problem. Because a broken DNA molecule is dangerous, the cell will attempt to repair it. If a DNA segment is snuck into the cell before the repair occurs, the cell can insert the new (and usually improved) DNA piece, providing a method to "edit" DNA.

The implications for such a technology are obvious. Such a method could be used, for example, to cure a person of a genetic disease or more easily produce genetically enhanced crops for farmers. But there are even cleverer uses. Because the CRISPR-Cas9 system can be designed to be self-propagating, it can be used to force a gene into a population of animals, such as mosquitoes. If this system targets genes that are important for survival or reproduction, then once released, this "gene drive" would rapidly spread through the population, killing off mosquitoes. (See infographic from The Economist.)

Now, a team of researchers writing in the journal Nature Communications has shown that a gene drive can be used to suppress infection with cytomegalovirus, a type of herpesvirus. The underlying molecular mechanism of the gene drive is similar to others before it: A self-propagating chunk of DNA inserts itself into a gene that is important to the virus. In this case, the gene is UL23, which is needed for cytomegalovirus to avoid the human immune response.

The researchers showed that when a cell is infected by both the normal virus (called "wildtype" or "WT") and the modified virus carrying a gene drive ("GD"), the gene drive was able to quickly and efficiently spread through the entire population, representing up to 95% of the final proportion of viruses. The end result is the suppression of viral infection (in cell culture, not in an animal model) because the gene drive virus lacks the important UL23 gene, which is needed for the virus to avoid a potent immune molecule known as interferon gamma(IFN-), which the authors added to the cell culture.

Could such a system work to treat viral infections in humans? Possibly. The authors note that a different gene (other than UL23) might need to be targeted, since lack of this gene is only fatal to the virus if IFN- is added to the cell culture. There are also concerns that a gene drive system could cause the viruses to mutate in various ways and may have unforeseen consequences.

Still, the technology is powerful and should be researched further. The coronavirus pandemic reminds us that we want to have multiple weapons in the public health arsenal should we be confronted with another life-threatening microbe.

Source: Walter, M., Verdin, E. Viral gene drive in herpesviruses. Nat Commun 11, 4884 (2020). Published: 28-Sept-2020. DOI: 10.1038/s41467-020-18678-0

More:
Gene Drives Could Kill Mosquitoes and Suppress Herpesvirus Infections - American Council on Science and Health

One summer day almost 20 years ago, a group of protestors arrived at a plot of genetically modified corn growing near the town of Montelimar in southern France.

They were led by Jos Bov, a left-wing activist famous for his skirmishes with the law and his tremendous moustache. Using machetes and shears, the protestors uprooted the crops and dumped the debris outside the offices of the regional government.

I thought about Bov this week as I read a new report on the next generation of genetic food technology. The techniques in the report make the processes that Bov opposed look clunky.

The GMOs he destroyed were created by inserting genes from other organisms say a stretch of DNA that confers resistance to a particular herbicide into a plants genome. This brute force approach is time-consuming and hard to control. Now scientists are using a new suite of gene-editing techniques, including a process known as CRISPR, to rapidly and precisely control the behavior of specific plant genes.

Gene-edited crops already exist. Scientists at the biotech firm Corteva, for example, have developed a high-yield strain of a variety of corn used in food additives and adhesives. Yet these initial advances belie the technologys potential.

Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones?

The power of gene editing can be wielded to modify plants and, among other things, achieve significant sustainability wins.

Here are a few potential outcomes explored in the new report, published by the Information Technology & Innovation Foundation, a pro-technology think tank:

This potential is thrilling, and there are signs that it will arrive soon. In China, where the government has made a big bet on gene-editing technology, numerous labs are working on crop strains that require less pesticides, herbicides and water. In the United States, a small but growing group of gene-editing startups is bringing new varieties to market, including an oilseed plant that can be used as a carbon-sequestering cover crop during the winter.

Yet when I read the ITIF report, I thought of Bov. Not because I agree with everything he said. Twenty years and many studies later, we know that the anti-GMO activists were wrong to say that modified crops posed a threat to human health. (The demonization of GMOs had profound consequences nonetheless: Fears about the risks posed by the crops are one reason why the crops are highly restricted in Europe and viewed warily by some consumers on both sides of the Atlantic.)

The reason I thought of Bov is that, at one level, he and other activists were pushing society to take a broader view of GMOs. They wanted people to ask who and what the crops were for, because they believed, rightly, that the crops were produced mainly with the profits of ag companies in mind.

Thats not to say its a bad thing for ag companies to be profitable. But our food systems affect so many aspects of our lives from the composition of the atmosphere to the prevalence of disease. When GMOs first began to be planted, there hadnt been enough debate about how the technology might affect these things. No wonder people were angry.

Thats a lesson I hope we can remember as gene editing shapes agriculture. Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones? If they can, we might end up with crops that everyone wants.

This article was adapted from the GreenBiz Food Weekly newsletter. Sign up here to receive your own free subscription.

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Get ready for the next wave of GMOs | Greenbiz - GreenBiz

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CRISPR in Agriculture Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

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Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

CRISPR & Cas Genes Market size 2020-2025 report, added by Market Study Report, unveils the current & future growth trends of this business sphere in addition to outlining details regarding the myriad geographies that form a part of the regional spectrum of CRISPR & Cas Genes market. Intricate details about the supply & demand analysis, contributions by the top players, and market share growth statistics of the industry are also elucidated in the report.

The research study on the CRISPR & Cas Genes market projects this industry to garner substantial proceeds by the end of the projected duration, with a commendable growth rate liable to be registered over the estimated timeframe. Elucidating a pivotal overview of this business space, the report includes information pertaining to the remuneration presently held by this industry, in tandem with a meticulous illustration of the CRISPR & Cas Genes market segmentation and the growth opportunities prevailing across this vertical.

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A brief run-through of the industry segmentation encompassed in the CRISPR & Cas Genes market report:

Competitive landscape:

Companies involved: CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc

Vital pointers enumerated:

The CRISPR & Cas Genes market report provides an outline of the vendor landscape that includes companies such as CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc. Parameters such as the distribution and sales area, alongside other pivotal details such as the firm profiling and overview have also been mentioned.

The study mentions the products manufactured by these esteemed companies as well the product price prototypes, profit margins, valuation accrued, and product sales.

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Geographical landscape:

Regions involved: USA, Europe, Japan, China, India, South East Asia

Vital pointers enumerated:

Segmented into USA, Europe, Japan, China, India, South East Asia, as per the regional spectrum, the CRISPR & Cas Genes market apparently covers most of the pivotal geographies, claims the report, which compiles a highly comprehensive analysis of the geographical arena, including details about the product consumption patterns, revenue procured, as well as the market share that each zone holds.

The study presents details regrading the consumption market share and product consumption growth rate of the regions in question, in tandem with the geographical consumption rate with regards to the products and the applications.

Product landscape

Product types involved: Vector-based Cas, DNA-free Cas and Cell Line Engineering

Vital pointers enumerated:

The CRISPR & Cas Genes market report enumerates information with respect to every product type among CRISPR Therapeutics, Genscrip, AstraZeneca, Caribou Biosciences, Inc., Cellectis, Addgene, F. Hoffmann-La Roche Ltd., Editas Medicine, Inc., Horizon Discovery Group Plc, Egenesis, Takara Bio, Inc., Mammoth Biosciences, Synthego, Danaher Corporation, Merck KGaA, Lonza, Cibus, New England BioLabs, Intellia Therapeutics, Inc., Inscripta and Inc, elaborating on the market share accrued, projected remuneration of each type, and the consumption rate of each product.

Application landscape:

Application sectors involved: Biotechnology and Pharmaceutical Companies, Academics and Government Research Institutes and Contract Research Organizations (CROs

Vital pointers enumerated:

The CRISPR & Cas Genes market report, with respect to the application spectrum, splits the industry into Biotechnology and Pharmaceutical Companies, Academics and Government Research Institutes and Contract Research Organizations (CROs, while enumerating details regarding the market share held by each application and the projected value of every segment by the end of the forecast duration.

The CRISPR & Cas Genes market report also includes substantial information about the driving forces impacting the commercialization landscape of the industry as well as the latest trends prevailing in the market. Also included in the study is a list of the challenges that this industry will portray over the forecast period.

Other parameters like the market concentration ratio, enumerated with reference to numerous concentration classes over the projected timeline, have been presented as well, in the report.

For More Details On this Report: https://www.marketstudyreport.com/reports/global-crispr-cas-genes-market-growth-status-and-outlook-2020-2025

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CRISPR & Cas Genes Market Analysis with Key Players, Applications, Trends and Forecasts to 2025 - The Daily Chronicle

DUBLIN--(BUSINESS WIRE)--The "Global Mice Model Market by Mice Type (Inbred, Knockout), Technology (CRISPR, TALEN, ZFN), Application (Oncology, Diabetes, Immunology), Service (Breeding, Cryopreservation, Genetic Testing), Care Products (Cages, Bedding, Feed), and Region - Forecast to 2025" report has been added to ResearchAndMarkets.com's offering.

The global mice model market size is projected to reach USD 1.9 billion by 2025 from USD 1.4 billion in 2020, at a CAGR of 6.4% during the forecast period.

The growth of this market is driven mainly by ongoing innovations in mice models, growing demand for personalized medicine, continuous support in the form of grants and investments, growth in the number of pharmaceutical R&D activities, and increasing focus of associations on the development of embryonic stem cells as well as knockout and mutant mice. Moreover, the popularity of humanized mice models and emerging technologies such as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) will present lucrative opportunities for the market in the coming years.

By mice type, the genetically engineered mice accounted for the fastest-growing segment of the mice model market

Genetically engineered mice segment is anticipated to be the fastest-growing due to the widespread use of these mice in diverse research areas, the emerging CRISPR technology, increasing focus on personalized medicine with the continuous introduction of new models

By service, the breeding segment accounted for the largest share of the mice model market

The breeding segment is expected to account for the largest market share in 2020, with the highest growth rate as well. This can primarily be attributed to the increasing demand for mice models for drug discovery and development and the subsequent increase in the demand for personalized medicines.

Oncology segment expected to grow at the fastest growth rate during the forecast period

Based on application, the mice model market has been segmented into oncology studies, immunology and inflammation studies, endocrine metabolic studies, cardiovascular studies, central nervous system studies (CNS), genetic studies, infectious disease studies, and other disease studies. The endocrine disease studies segment is further segmented into diabetes and other endocrine metabolic disease. The oncology segment is expected to account for the largest market share in 2020, with the highest growth rate as well. This can primarily be attributed to the increasing number of patients who have cancer and the subsequent increase in the demand for cancer therapies.

By technology type, CRISPR/Cas9 accounted for the largest share of the mice model market

CRISPR is the most widely used technology in the mice model market and contributed to the largest share of the mice model market in 2020. Ease of design, high efficiency, and relatively lower cost have increased the demand for CRISPR-customized mice models.

By mice care product, cages segment accounted for the largest share of the mice model market

Based on mice care products, the mice model market has been segmented into cages, feed, bedding, and other products (gnotobiotic equipment, water systems, and accessories). The cages segment accounted for the largest share of the mice model market. This can be attributed to the availability of a wide range of cages designed for specific research needs and the higher cost of cages compared to other care products.

Asia Pacific: The fastest-growing region in the mice model market

The Asia Pacific market is projected to grow at the highest CAGR during the forecast period. Several global pharmaceutical firms have entered the APAC market to tap the significant growth opportunities in emerging Asian countries and lower their production costs by shifting their drug discovery R&D operations and manufacturing to the region. A large number of qualified researchers and low-cost operations in APAC countries, such as India and China, are some of the major factors supporting this trend.

North America: The largest share of the drug discovery services market

North America, which includes the US and Canada, accounted for the largest share of the mice model market. The large share of the North America region can be attributed to the presence of major players operating in the mice model market in the US, growing biomedical research in the US, and rising preclinical activities by CROs and pharmaceutical companies in the region.

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/17ab4p

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$1.9 Billion Mice Model Market by Mice Type, Technology, Application, Service, Care Products - Global Forecast to 2025 - ResearchAndMarkets.com -...

Timothy Ray Brown, who became the first HIV patient to be cured of the infection, died September 29 of leukemiathe very disease that led to the fortuitous eradication of the virus from his body. He was 54.

Until he disclosed his identity, Brown was known as the Berlin patient, whose HIV infection was eliminated in 2007 after undergoing a stem cell transplant to treat acute myeloid leukemia. The bone marrow donor was selected to have a naturally occurring genetic variant that blocked HIV from entering cells. The treatment workedboth for his cancer, and his viral infection.

Timothy symbolized that it is possible, under special circumstances to cure HIV, Gero Htter, the doctor who performed the stem cell transplant, tells theAssociated Press.

Until2016, Brown remained the only person in the world to have been cured of AIDS using this approach and his unique experience motivated him to advocate for AIDS research. As he toldThe Scientist in 2015, I didnt want to be the only one in my club.

Brown was born in 1966 and grew up in Seattle. He was living in Berlin when he received the diagnosis of leukemia and sought treatment from Htter. The doctor had previously read about individuals with variants in the CCR5 gene, which codes for a receptor on cell surfaces, that gives themnatural immunity to HIV. Upon finding out that Brown was HIV-positive, Htter decided to look for a bone marrow donor who might have this variant. As Htter explained to The Scientist in 2015, he screened dozens of donors until he found one with the so-called delta32 mutation.

Within months of the transplant, the virus was gone from Browns cells, although his recovery was difficult and he required a second transplant to treat the leukemia.

In 2012, Brown and activist Dave Purdy started the Cure for AIDS Coalition to raise awareness of HIV research. According to aFacebook post by Browns partner, Tim Hoeffgen, Tim committed his lifes work to telling his story about his HIV cure and became an ambassador of hope. Tim also gave numerous blood and tissue samples to researchers after his cure.

The invasiveness of the bone marrow transplant precludes it from being applied more widely to HIV patients, but the insights gained from Browns successful cure have inspired further work on CCR5. For instance, in 2017, researchers used CRISPR to disrupt the gene in human hematopoietic stem cells anddemonstrated that these cells could ward off HIV infection in mice transplanted with them. More recently, andcontroversially, the gene was a target of CRISPR-based editing in human embryos to make them resistant to HIV.

Brown never again tested positive for HIV. His leukemia, however, relapsed five months ago.

Timothy was a champion and advocate for keeping an HIV cure on the political and scientific agenda, Sharon Lewin, the director of the Doherty Institute in Melbourne, Australia, tells theBBC. It is the hope of the scientific community that one day we can honour his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure using gene editing or techniques that boost immune control.

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Timothy Ray Brown, First Person to Be Cured of HIV, Dies - The Scientist

The comprehensive research report on theCRISPR and Cas Genes Marketinfluences iterative and comprehensive research methodology to offer insights into the existing market scenario over the forecast timeframe. The report also delivers in-depth details about the growth and development trends that will have a major impact on the behavior of the CRISPR and Cas Genes market in the approaching years. Furthermore, the report touches upon other key pointers such as the regional aspects and policies overriding the industry. The report suggests that the global CRISPR and Cas Genes market Demand is expected to witness a considerable CAGR growth of % during the forecast period and surpass the value of ~US$ by 2026.

Report Covers Impacts of COVID-19 to the market.

The on-going pandemic has overhauled various facets of the market. This research report provides the financial impacts and market disturbance on the CRISPR and Cas Genes market. It also includes analysis on the potential lucrative opportunities and challenges in the foreseeable future. Fact.MR has interviewed various delegates of the industry and got involved in the primary and secondary research to confer the clients with information and strategies to fight against the market challenges amidst and after COVID-19 pandemic.

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Market Segmentation:

Few of the companies that are covered in the report,

Note: Additional companies can be included in the list upon the request.

On the basis of product,

On the basis of end use,

On the basis of region, the CRISPR and Cas Genes market study contains:

The research report provides a detailed analysis of the prominent player in the market, products, applications, and regional analysis which also include impacts of government policies in the market. Moreover, you can sign up for the yearly updates on the CRISPR and Cas Genes market.

Key Questions Answered in this Report on the CRISPR and Cas Genes Market:

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Reports published by Fact.MR are a result of the combination of our experts and digital technologies. We thrive to provide innovative business solutions to the clients as well as tailor the reports aligning with the clients requisites. Our analysts perform comprehensive research to offer ins and outs of the current market situation. Clients across various time zones tend to utilize our 24/7 service availability.

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Demand Predicted for CRISPR and Cas Genes Market, COVID-19 Pandemic puts Existing Projections in Jeopardy Fact.MR Report - The Cloud Tribune

In the Patent Trial and Appeal Board's decision on motions issued September 10th in Interference No. 106,115 (see"PTAB Decides Parties' Motions in CRISPR Interference")between Senior Party The Broad Institute, Harvard University, and the Massachusetts Institute of Technology (collectively, "Broad") and Junior Party the University of California/Berkeley, the University of Vienna, and Emmanuelle Charpentier (collectively, "CVC") the Board denied Broad's Motion No. 2 to substitute the Count.

To recap, the Count in the '115 interference as declared recited in the alternative either claim 18 of the Broad's U.S. Patent No. 8,697,359 (dependent on claim 15), which taken together recites the following invention:

An engineered, programmable, non-naturally occurring Type II CRISPR-Cas system comprising a Cas9 protein and at least one guide RNA that targets and hybridizes to a target sequence of a DNA molecule in a eukaryotic cell, wherein the DNA molecule encodes and the eukaryotic cell expresses at least one gene product and the Cas9 protein cleaves the DNA molecules, whereby expression of the at least one gene product is altered; and, wherein the Cas9 protein and the guide RNA do not naturally occur together,wherein the guide RNAs comprise a guide sequence fused to a tracr sequence.

(where the underlined portion recites the relevant language from claim 18), or Claim 156 of Berkeley's U.S. Patent Application No. 15/981,807:

A eukaryotic cell comprising a target DNA molecule and an engineered and/or non-naturally occurring Type II Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)- CRISPR associated (Cas) (CRISPR-Cas) system comprisinga) a Cas9 protein, or a nucleic acid comprising a nucleotide sequence encoding said Cas9 protein; andb) a single molecule DNA-targeting RNA, or a nucleic acid comprising a nucleotide sequence encoding said single molecule DNA-targeting RNA; wherein the single molecule DNA-targeting RNA comprises: i) a targeter-RNA that is capable of hybridizing with a target sequence in the target DNA molecule, and ii) an activator-RNA that is capable of hybridizing with the targeter-RNA to form a double-stranded RNA duplex of a protein- binding segment,wherein the activator-RNA and the targeter-RNA are covalently linked to one another with intervening nucleotides; andwherein the single molecule DNA-targeting RNA is capable of forming a complex with the Cas9 protein, thereby targeting the Cas9 protein to the target DNA molecule, whereby said system is capable of cleaving or editing the target DNA molecule or modulating transcription of at least one gene encoded by the target DNA molecule.

Broad's Motion No. 2 requested that the Board substitute proposed Count 2:

A method, in a eukaryotic cell, of cleaving or editing a target DNA molecule or modulating transcription of at least one gene encoded by the target DNA molecule, the method comprising:contacting, in a eukaryotic cell, a target DNA molecule having a target sequence with an engineered and/or non-naturally-occurring Type II Clustered Regularly lnterspaced Short Palindromic Repeats (CRISPR)-CRISPR associated Cas) (CRISPR-Cas) system comprising: a) a Cas9 protein, and b) RNA comprising i) a targeter-RNA that is capable of hybridizing with the target sequence of the DNA molecule or a first RNA comprising (A) a first sequence capable of hybridizing with the target sequence of the DNA molecule and (B) a second sequence; and ii) an activator-RNA that is capable of hybridizing to the targeter-RNA to form an RNA duplex in the eukaryotic cell or a second RNA comprising a tracr sequence that is capable of hybridizing to the second sequence to form an RNA duplex in the eukaryotic cell,wherein, in the eukaryotic cell, the targeter-RNA or the first sequence directs the Cas9 protein to the target sequence and the DNA molecule is cleaved or edited or at least one product of the DNA molecule is altered.

The distinction Broad made was between embodiments of CRISPR methods that are limited to "single-molecule guide RNA" (aka "fused" or "covalently linked" species), versus embodiments that encompass single-molecule and "dual molecule" species (wherein in the latter versions, the "targeter-RNA" and "activator-RNA" as recited in the proposed Count are not covalently linked). Broad argued that its Proposed Count 2 should be adopted by the Board because it "properly describes the full scope of the interfering subject matter between the parties because both parties have involved claims that are generic, non-limited RNA claims." The brief also argued that Proposed Count 2 "sets the correct scope of admissible proofs [i.e., their own] for the breakthrough invention described by the generic claims at issue in these proceedingsthe successful adaption of CRISPR-Cas9 systems for use in eukaryotic environments," which Broad contended current Court 1 (in either alternative) does not.

The Board denied this motion for the simple reason that, in its opinion, "Broad fails to provide a sufficient reason why the count should be changed." Citing Louis v. Okada, 59 U.S.P.Q.2d 1073, 1076 (BPAI 2001) (relied upon in opposition by CVC), the Board notes that it will only change the Count when reasons for doing so are "compelling." Broad's motion argued that their claims (and CVC's) were directed to eukaryotic embodiments of CRISPR that were not limited to either single- or dual-molecule RNA species, but that the phrase "guide RNA" was generic. Based on the claim construction, the Board rejected this construction, limiting the claims to single-molecule RNA embodiments.

The Decision also states that "Broad's argument for broadening the scope of the count to be generic as to RNA configuration is unpersuasive." According to the Decision, CVC convinced the Board that there were other differences between Count 1 (as declared in the interference) and Broad's proposed Count 2. These include that Count 2 is directed to a method whereas Count 1 recites system or eukaryotic cell. This is enough, the Board states, for the PTAB to deny Broad's Motion No. 2 simply on these grounds. The Board also was persuaded by CVC's argument that all of the Broad's claims are directed to "guide RNA" or "chimeric RNA" and thus to single-RNA molecule eukaryotic CRISPR embodiments. Further, the Board faulted the Broad for not specifically identifying all the claims it contends recite generic eukaryotic CRISPR embodiments with regard to its RNA components. Continuing, the Decision asserts that Broad also failed to convince the Board that the few claims that expressly recited "fused" RNA embodiments were sufficient under the doctrine of claim differentiation to construe the independent claims as encompassing both single- and dual-RNA molecule eukaryotic CRISPR embodiments.

As is its wont, the Board identified formal deficiencies in some Broad arguments that were sufficient to deny the relief requested under the rubric set forth in 37 C.F.R. 41.121(b) that "the party filing the motion has the burden of proof to establish that it is entitled to the requested relief." These include instances where the Broad's brief cited a footnote that does not stand for the cited proposition, and hence that "CVC did not have notice of arguments regarding claim 15 or of any other claim Broad asserts is directed to a generic RNA configuration without using the term 'guide RNA'". Accordingly, the Board concluded that "[b]ecause Broad did not provide arguments about the interpretation of specific claims in its Motion 2 we are not persuaded by its argument that the scope of the 'vast majority' of its claims requires a broader count."

The Board's Decision also turns on its head the Broad's argument (recited throughout its briefing) that this interference is unfair to Broad due to "CVC's strategic decisions" in earlier Interference No. 105,048 between the parties. The Board notes that the outcome in that interference, that there was no interference-in-fact, "achiev[ed] Broad's desired remedyending the interference." "Had Broad wished to remain in a priority contest with CVC under the count in that interference, it could have chosen not to file the motion for no interference-in-fact," according to the decision, and thus the Board saw "no unfairness in Broad not having had a chance to present its best proofs in a priority contest with CVC in the '048 interference under these circumstances."

This portion of the decision concludes by denying Broad's alternative remedy of redeclaring the interference with both Counts, the Board stating its reasoning that "Broad fails to explain why this would be an appropriate remedy, given that we are not persuaded that a majority, or even a significant number, of its claims are drawn to a generic RNA configuration."

The remainder of the Board's Decision will be discussed in future posts.

Continue reading here:
PTAB Denies Broad Motion No. 2 to Substitute the Interference Count - JD Supra

SAN FRANCISCO, Sept. 28, 2020 /PRNewswire/ -- SynBioBeta, the leading community of biological engineers, investors, innovators, and entrepreneurs to build a betterworld with biology, announced the schedule for its 2020 Global Synthetic Biology Summit.

The Summit will feature such luminaries as Tristan Harris (Center for Humane Technology), George Church (Harvard), Jennifer Holmgren (LanzaTech), Christina Smolke (Antheia), Sylvia Wolf (AquaBounty), Ed Boyden (MIT), and Timothy Lu (MIT).

Despite the economic slowdown of COVID, synthetic biology startups raised arecord-setting $3.0 billion in the first half of 2020. While funding is strong for tools and technologies companies -- the engine of thebioeconomy-- there is increasing investment in synthetic biology-enabled companies in consumer products, food, agriculture, medicine, chemicals, materials, and other manufacturing sectors, signaling the impact techand biology is poised tohave on every industry.

"This year, the pandemic has brought previously unimaginable challenges to ourcommunity, not just how we meet and work, but more importantly, how we respondto the society's urgent needs," said John Cumbers, founder and CEO of SynBioBeta,which earlier in the year hosted a series of events on synthetic biology and thepandemic. "Synthetic biology is ready to turn today's industry on its head and revolutionize the way we do business. In the same way that every company today isin some wayan Internet company, every company will one day be a biology company. SynBioBeta 2020 is the place to get ahead of the curve."

This year's conference will explore how engineered biology will disrupt consumer products, food, agriculture, medicine, chemicals, materials, and more. Sessions include:

Each year, SynBioBeta is honored to recognize synthetic biology leaders whoembody the best of this industry and the aims it seeks to achieve. This year'swinners are an exceptional group of innovators who have helped the communitygrow while making profound contributions to society:

About SynBioBeta 2020SynBioBeta 2020 is the Global Synthetic Biology Conference that unites leadingbiological engineers, investors, innovators, and entrepreneurs who are building the future with biology. This year's digital offering gives you even more ways to connect,including our annual conference, new events and grand challenges, access to online contentand groups, and AI-powered networking. Learn the latest technologies, hear the big announcements in the field, make new partnerships, meet investors, and discover new companies. Learn more and register here.

About SynBioBetaSynBioBeta is the leading community of innovators, investors, engineers, andthinkers who share a passion for using synthetic biology to build a better, more sustainable universe. We create and energize innovation communities to make theimpossible possible via unparalleled opportunities for growth, networking, storytelling, and learning.

SynBioBeta offers a weekly industry digest, The Bioeconomy Hub membershipprogram, the SynBioBeta Podcast, Good Genes magazine, and educationalcourses in addition to providing our world-class industry partners with opportunities for advertising, partnership, trade show exhibition, strategic consultation, and promotion.

For more information, visit http://www.synbiobeta.com.

Contact: Amanda Prieto, [emailprotected], (707) 344-8279

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nobel-laureate-frances-arnold.jpg Nobel Laureate Frances Arnold Receives 2019 SynBioBeta Award Nobel Laureate Frances Arnold received 2019 SynBioBeta Award from SynBioBeta founder John Cumbers

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Synthetic Biology Industry Gathers at SynBioBeta 2020 Global Summit to Grow the Bioeconomy, Fight the Pandemic, and Honor CRISPR Pioneer Jennifer...

Here's a helpful hint for you: Ignore the market volatility. If you watch your stocks continually, the up-and-down swings could make you a nervous wreck. Invest with a long-term perspective, and the temporary market gyrations will be much less bothersome.

The good news is that there are plenty of stocks that provide opportunities to deliver excellent long-term returns. Here are three stocks that could even double your money within the next few years.

Image source: Getty Images.

CRISPR Therapeutics (NASDAQ:CRSP) isn't too far away from doubling this year, with its shares up close to 80%. But the biotech stock should still have plenty of room to run.

You'll want to especially watch CRISPR Therapeutics' lead pipeline candidate, CTX001. CRISPR and its big partner Vertex Pharmaceuticalsare currently evaluating the gene-editing therapy in early stage clinical studies targeting rare blood diseases beta-thalassemia and sickle cell disease. The companies have already reported encouraging preliminary results from these studies and expect to announce additional data in the next few months. CTX001 holds the potential to essentially cure these diseases.

CRISPR Therapeutics also has a big opportunity with its three allogeneic chimeric antigen receptor T-cell (CAR-T) therapies in early stage clinical testing. Allogeneic CAR-T therapies use immune cells called T-cells from healthy individuals that are genetically engineered to target specific types of cancer then infused into sick patients. Current CAR-T therapies on the market use the sick patients' own T-cells -- an approach that's a lot slower and costlier than allogeneic therapies should be. CRISPR expects to report results from its early stage trial of one its CAR-T therapies, CTX110, by the end of this year.

Sure, there's a real risk that CRISPR Therapeutics' gene-editing programs will flop. But so far, investors have plenty of reasons to be cautiously optimistic. If CTX001 or any of the company's allogeneic CAR-T therapies succeed in clinical testing, CRISPR Therapeutics should easily double in the not-too-distant future.

Fastly (NYSE:FSLY) has taken investors on a really wild ride so far in 2020. The tech stock has had seven swings of at least 20% in just the last three months. But even with the turbulence, Fastly is still up more than 350% year to date.

The company's name hints at its underlying business. Fastly focuses on speeding up the delivery of apps and data over the internet through content delivery networks (CDNs) and edge computing. CDNs reduce the physical distance between servers and end users with a widespread distributed platform of servers. Edge computing takes a somewhat similar approach by moving app and data processing close to the cloud's edge -- the point where corporate networks connect with the cloud.

Fastly's addressable market currently totals more than $35 billion. It could grow much bigger than that with the unstoppable migration of apps to the cloud. Fastly has less than 1% of this market right now, giving the company a massive growth opportunity.

The company's biggest customer is TikTok, the video-sharing social network that has been at the center of a political firestorm recently. Until a deal is completely finalized to separate TikTok from its China-based owner ByteDance, Fastly's shares could remain highly volatile. However, Fastly's long-term prospects look great.

If you have any doubts about how hot the U.S. cannabis market is, just look atInnovative Industrial Properties (NYSE:IIPR). IIP's shares have soared more than 60% so far this year and have skyrocketed close to 600% over the last three years.

IIP ranks as the leading real estate investment trust (REIT) focused on the medical cannabis industry. It buys properties from medical cannabis operators, then turns around and leases the properties back to the operators. This helps tenants raise cash while giving IIP a steady revenue stream.

The way for IIP to double is pretty simple: Keep doing what it has been doing. IIP currently owns 63 medical cannabis properties in 16 states, up from 46 properties in 14 states at the end of 2019. The company's attractive dividend, which currently yields nearly 3.8%, makes it even easier for IIP to deliver tremendous returns for investors.

It's possible that the legalization of marijuana at the federal level in the U.S. could pave the way for more rivals to enter IIP's market. However, marijuana legalization isn't a slam dunk by any means. And even if IIP faces increased competition in the future, the growth in the overall U.S. cannabis market should enable this stock to continue its winning ways.

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3 Stocks That Could Double Your Money - The Motley Fool

Dublin, Sept. 30, 2020 (GLOBE NEWSWIRE) -- The "Global Precision Medicine Market: Focus on Ecosystem, Technology, Application, Country Data (21 Countries), and Competitive Landscape - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

Global Precision Medicine Market to Reach $278.61 Billion by 2030

Precision medicine refers to the medicine developed as per an individual's genetic profile. It provides guidance regarding the prevention, diagnosis, and treatment of diseases. The segmentation of the population is done depending on the genome structure of the individuals and their compatibility with a specific drug molecule.

In the precision medicine market, the application of molecular biology is to study the cause of a patient's disease at the molecular level, so that target-based therapies or individualized therapies can be applied to cure the patient's health-related problems.

This industry is gaining traction due to the increasing awareness about healthcare among individuals, integration of smart devices such as smartphones and tablets into healthcare, and increasing collaborations and agreements of IT firms with the diagnostics and biopharmaceutical companies for the development of precision diagnostic tools.

Within the research report, the market is segmented on the basis of product type, ecosystem application, and region, which highlight value propositions and business models useful for industry leaders and stakeholders. The research also comprises country-level analysis, go-to-market strategies of leading players, future opportunities, among others, to detail the scope and provide 360-degree coverage of the domain.

Key Topics Covered:

1 Product Definition

2 Research Scope

3 Research Methodology

4 Global Precision Medicine Market Overview4.1 Market Definition4.2 Precision Medicine: A Frontier in the Genesis of Patient-centric Medicine4.3 Precision Medicine: Remodeling the One-Size-Fits-All Theory to Individually Tailored Therapy4.4 Initiatives and Programs4.5 Precision Medicine: Enabling Technologies and Applications4.5.1 Innovators4.5.1.1 3D DNA Printing4.5.1.1.1 Introduction4.5.1.1.2 Role of 3D DNA Printing4.5.1.2 RNA-Seq4.5.1.2.1 Introduction4.5.1.2.2 Role of RNA-Seq in Precision Medicine4.5.1.2.3 Key Players4.5.1.3 4D Molecular Imaging4.5.1.3.1 Introduction4.5.1.3.2 Role of 4D Molecular Imaging in Precision Medicine4.5.1.3.3 Key Players4.5.2 Early Adopters4.5.2.1 CRISPR4.5.2.1.1 Introduction4.5.2.1.2 Role of CRISPR in Precision Medicine4.5.2.1.3 Key Players4.5.2.2 Blockchain4.5.2.3 Imaging Informatics4.5.3 Early Majority4.5.3.1 Artificial Intelligence (AI)4.5.3.2 Circulating Free DNA (cfDNA)4.5.3.3 Big Data4.5.3.4 Next-Generation Sequencing (NGS)4.5.3.5 Health Informatics4.5.3.6 Bioinformatics4.5.4 Late Majority4.5.4.1 Polymerase Chain Reactions (PCR)4.5.4.2 Microarray4.6 COVID-19 Impact on the Global Precision Medicine Market

5 Market Dynamics5.1 Overview5.2 Market Drivers5.2.1 Advancement of Sequencing Technologies5.2.2 Rising Prevalence of Chronic Diseases5.2.3 Growing Demand for Preventive Care5.2.4 Shifting the Significance in Medicine, from Reaction to Prevention5.2.5 Reducing Adverse Drug Reactions Through Pharmacogenomics Test5.2.6 Potential to Reduce the Overall Healthcare Cost Across the Globe5.3 Market Restraints5.3.1 Unified Framework for Data Integration5.3.2 Limited Knowledge about Molecular Mechanism/ Interaction5.3.3 Lack of Robust Reimbursement Landscape5.3.4 Regulatory Hurdles5.4 Market Opportunities5.4.1 Targeted Gene Therapy5.4.2 Expansion into the Emerging Markets5.4.3 Collaboration and Partnerships Across Value Chain to Accelerate the Market Entry

6 Industry Insights6.1 Patent Analysis6.2 Legal Requirements and Regulations6.3 Pipeline Analysis6.4 Legal Requirements and Framework by the FDA6.5 Legal Requirements and Framework by the EMA6.6 Legal Requirements and Framework by the MHLW

7 Competitive Landscape7.1 Synergistic Activities7.1.1 Product launches, Enhancements, and Upgradation7.1.2 Product Approvals7.1.3 Mergers and Acquisitions7.1.4 Business Expansion7.2 Market Share Analysis7.2.1 Market Share Analysis by Applied Sciences, 20197.2.2 Market Share Analysis by Precision Diagnostics, 20197.2.3 Market Share Analysis by Precision Therapeutics, 20197.2.4 Market Share Analysis by Digital Health and IT, 2019

8 Global Precision Medicine Market (by Ecosystem)8.1 Overview8.2 Applied Sciences8.2.1 Genomics8.2.2 Global Precision Medicine Genomics Market (by Technology)8.2.2.1 Polymerase Chain Reaction (PCR)8.2.2.2 Next-Generation Sequencing (NGS)8.2.2.3 Genome Editing8.2.2.4 Other Technologies8.2.3 Pharmacogenomics8.2.4 Other Applied Sciences8.3 Precision Diagnostics8.3.1 Molecular Diagnostics (MDx)8.3.2 Global Precision Medicine Molecular Diagnostics Market (by Type)8.3.2.1 Non-Invasive Prenatal Testing (NIPT)8.3.2.2 Companion Diagnostics8.3.2.3 Liquid Biopsy8.3.2.4 Other Molecular Diagnostics8.3.3 Medical Imaging8.3.3.1 Global Precision Medicine Medical Imaging Market (by Type)8.3.3.1.1 Imaging Analytics8.3.3.1.2 Imaging Computer-Aided Detection (CADx)8.3.3.2 Global Precision Medicine Medical Imaging Market (by Region)8.4 Digital Health and Information Technology8.4.1 Global Precision Medicine Digital Health and Information Technology Market (by Type)8.4.1.1 Clinical Decision Support Systems (CDSS)8.4.1.2 Big Data Analytics8.4.1.3 IT Infrastructure8.4.1.4 Genomics Informatics8.4.1.5 In-Silico Informatics8.4.1.6 Mobile Health8.5 Precision Therapeutics8.5.1 Global Precision Medicine Therapeutics Market (by Type)8.5.1.1 Clinical Trials8.5.1.2 Cell Therapy8.5.1.3 Drug Discovery and Research8.5.1.4 Gene Therapy

9 Global Precision Medicine Market (by Application)9.1 Overview9.2 Oncology9.2.1 Cancer Precision Medicine Drugs and Indications9.3 Infectious Diseases9.3.1 Infectious Diseases Precision Medicine Drugs and Indications9.4 Neurology9.4.1 Neurology Precision Medicine Drugs and Indications9.5 Cardiovascular9.5.1 Cardiovascular Precision Medicine Drugs/Tests/ and Indications9.6 Lifestyle and Endocrinology9.6.1 Endocrinology Precision Medicine Drugs and Indications9.7 Gastroenterology9.7.1 Gastroenterology Precision Medicine Drugs and Indications9.8 Other Applications9.8.1 Precision Drugs for Other Applications

10 Global Precision Medicine Market, (by Region)

11 Company Profiles11.1 Company Overview11.2 Role of Abbott Laboratories in Global Precision Medicine Market11.3 Financials11.4 Key Insights About Financial Health of the Company

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Global Precision Medicine Market 2020-2030: Remodeling the One-Size-Fits-All Theory to Individually Tailored Therapy - GlobeNewswire

The report is an all-inclusive research study of the global CRISPR And CRISPR-Associated (Cas) Genes Market taking into accounts the growth factors, recent trends, developments, opportunities, and competitive landscape. The CRISPR And CRISPR-Associated (Cas) Genes Market analyst and researchers have done a wide analysis of the global CRISPR And CRISPR-Associated (Cas) Genes Market with the help of research methodologies such as PESTLE and Porters Five Forces breakdown. They have provided exact and consistent market data and useful recommendations with an aim to help the players gain an insight into the overall current and upcoming market scenario.

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Leading manufacturers of CRISPR And CRISPR-Associated (Cas) Genes Market:

Caribou BiosciencesAddgeneCRISPR THERAPEUTICSMerck KGaAMirus Bio LLCEditas MedicineTakara Bio USAThermo Fisher ScientificHorizon Discovery GroupIntellia TherapeuticsGE Healthcare Dharmacon

The report presents a detailed competitive landscape along with a comprehensive analysis of the market share and size, product range, product innovation, technological advancements, and market patterns. The CRISPR And CRISPR-Associated (Cas) Genes Market report incorporates the study of recent developments in the market, such as product launches, mergers, acquisitions, collaborations, joint ventures, and partnerships, among others. The report offers a futuristic outlook of the market scenario for the forecast period of 2020-2026. The CRISPR And CRISPR-Associated (Cas) Genes Market regional analysis covers North America, Europe, Latin America, Asia-Pacific, and Middle East & Africa.

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CRISPR And CRISPR-Associated (Cas) Genes Market can be segmented into Product Types as

Genome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

CRISPR And CRISPR-Associated (Cas) Genes Market can be segmented into Applications as

Biotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

To breakdown the huge study that spreads through geographies, products, and end-use segments, among other market-specific segments, the authors present the CAGR of each segment during the years of anticipate. CAGR is a simplistic illustration of enlargement that visibly projects which segment register the highest/least growth through the forecast period 2020-2026.

The newest report about the CRISPR And CRISPR-Associated (Cas) Genes market provides a thorough estimate of the business vertical in question, alongside a brief overview of the industry segments. An extremely workable estimation of the present industry scenario has been delivering in the study, and the CRISPR And CRISPR-Associated (Cas) Genes market size with regards to the revenue and volume have also been mention.

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Highlights of the Report

Table of Contents:

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CRISPR And CRISPR-Associated (Cas) Genes Market Comprehensive Study Explores Huge Growth with Demand by Forecast 2024 - The Market Records

Global Genome Editing Market: Overview

Also known as genome editing with engineered nucleases (GEEN), genome editing is a method of altering DNA within a cell in a safe manner. The technique is also used for removing, adding, or modifying DNA in the genome. By thus editing the genome, it is possible to change the primary characteristic features of an organism or a cell.

The global genome editing market can be segmented on the basis of delivery method, technology, application, and geography. By technology, the global genome editing market can be segmented into Flp-In, CRISPR, PiggyBac, and ZFN. Based on delivery method, in vivo and ex vivo can be the two broad segments of the global genome editing market. By application, the global genome editing market can be categorized into medicine, academic research, and biotechnology.

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Global Genome Editing Market: Key Trends

Since genome editing is gaining rising adoption in the domain of scientific research for attaining a better understanding of biological aspects of organisms and how they work, the global genome editing market is likely to promise considerable growth over the forthcoming years. More importantly, genome editing is being used by medical technologies, where it can be used for modifying human blood cells which can then be placed back in the body for treating conditions such as AIDS and leukemia. The technology can also be potentially utilized to combat infections such as MRSA as well as simple genetic disorders including hemophilia and muscular dystrophy.

Global Genome Editing Market: Market Potential

As more easy-to-use and flexible genome technologies are being developed, greater potential of genome editing is being recognized across bioprocessing and treatment modalities. For instance, in May 2017, MilliporeSigma announced that it successfully developed a novel genome editing tool which can make the CRISPR system more productive, specific, and flexible. The researchers thus have a more number of experimental options along with faster results.

All this can lead to a growing rate of drug development, enabling access to more advanced therapies. Proxy-CRISPR, the new technique, makes access to earlier inaccessible aspects of the genome possible. As most of the existing CRISPR systems cannot manage without re-engineering of human cells, the new method is expected to gain more popularity by virtue of the elimination of the need for re-engineering, simplifying the procedures.

Several other market players are focusing on clinical studies with a view to produce effective treatments for different health conditions. For example, another major genome editing firm, Editas Medicine, Inc. announced the results of its pre-clinical study displaying the success of the CEP290 gene present in the retina of primates in the same month. With the positive results of the study, the companys belief in the vast potential of its candidate in the treatment of a genetically inherited retinal degenerative disease, Leber congenital amaurosis type 10, affecting childrens eyesight has been reinforced.

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Global Genome Editing Market: Regional Outlook

By geography, the global genome editing market can be segmented into Latin America, Europe, Asia Pacific, the Middle East and Africa, and North America. North America registered the highest growth in the past, and has been claiming the largest portion of the global genome editing market presently. The extraordinary growth of this region can be attributed to greater adoption of cutting edge technologies across several research organizations. The U.S., being the hub of research activities, is expected to emerge as the leading contributor. Asia Pacific is also likely to witness tremendous demand for genome editing over the forthcoming period, assisting the expansion of the global genome editing market.

Global Genome Editing Market: Competitive Analysis

CRISPR THERAPEUTICS, Caribou Biosciences, Inc., Sigma Aldrich Corporation, Sangamo, Intellia Therapeutics, Inc., Editas Medicine, Thermo Fisher Scientific, Inc., and Recombinetics, Inc are some of the key firms operating in the global genome editing market.

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Genome Editing Market Poised to Expand at a Robust Pace Over 2025 - The Daily Chronicle

On September 10, 2020, the Patent Trial and Appeal Board (PTAB) decided key motions in Interference No. 106,115,University of California v. Broad Institute. The interference involves 10 patent applications of the University of California (The Regents of the University of California, University of Vienna, and Emmanuelle Charpentier, collectively referred to as "UC") and 13 patents and one patent application of Broad Institute (The Broad Institute, Massachusetts Institute of Technology, and Presidents and Fellows of Harvard College, collectively referred to as "Broad"), all directed to the use of CRISPR-Cas9 to edit eukaryotic genomes. Broad and UC had argued these motions before the PTAB in May 2020.1

This decision is the latest turn in a long battle between Broad and UC. In a previous round of interference, Broad was able to secure its U.S. patents covering CRISPR-Cas9 in eukaryotes. The current interference puts these patents in question again. If UC prevails, Broad would lose its CRISPR-Cas9 eukaryote patents. Notably, UC also owns patents generally directed to CRISPR-Cas9 and not particularly to eukaryotes, which are not involved in this interference. Even if UC loses this interference, its general CRISPR-Cas9 patent claims would remain unaffected.

The PTAB decision recognized Broad as having the earliest effective filing date, but turned down its request to call off the current interference altogether. While the former gives Broad an advantage in proving it invented first, the latter appears to be a significant setback. The decision thus does not settle the dispute, but rather moves it to the next stage, where each party may submit actual evidence, such as lab notebooks, to prove they invented the disputed CRISPR-eukaryote system first. This stage may be rather long and complicated, and the parties may face increased pressure to settle.

A patent interference is a proceeding formerly used to determine who first invented a claimed invention. Interferences were phased out in a 2012 legislation, but patents or applications with effective filing dates before March 2013 still can be subject to an interference. To simplify the proofs of first invention, the disputed invention is defined in a "count" based on the parties' claims.

Details

Interference No. 106,115 is the second interference between Broad and UC over the CRISPR-Cas9 technology. The first interference between the parties ended in 2018 after an appeal, with the conclusion that the parties' claims did not in fact interfere because Broad's invention, directed to CRISPR-Cas9 in eukaryotic cells, would not have been obvious in light of UC's invention, which claims the CRISPR-Cas9 system generically. The current interference was triggered by new claims UC filed shortly after conclusion of the first interference, which have essentially the same scope as Broad's claims that survived the first interference. The patent examiner decided the new UC claims were allowable except for a potential interference with Broad's claims, and the PTAB subsequently declared the second interference on June 24, 2019.

The PTAB's decision on September 10 mainly addressed three issues: 1) whether the PTAB should recognize either party's first provisional application as its earliest effective filing date; 2) whether the first interference as affirmed by the Federal Circuit should bar or "estop" the current interference; and 3) whether Broad's request for a broader "count" should be granted.

Regarding the earliest effective filing date, each party requested the benefit of its first provisional application. Having the earliest recognized filing date makes one party the "senior party," which means the party is presumed to be the first inventora big advantage in a priority contest. Any other party would be a "junior party." In the current decision, the PTAB found that Broad's first provisional application, filed December 12, 2012, provided a constructive reduction to practice of an embodiment within the count, and UC's request for benefit to its first priority date was denied. The PTAB held that UC's earlier applications lacked discussion of PAM sequences, sample target DNA sequences, and special instructions or conditions necessary to accommodate the eukaryotic cellular environment. The PTAB found that these failings were not overcome until UC's third provisional application, which was filed on January 28, 2013. Accordingly, the PTAB redeclared the interference with Broad as the senior party and UC as the junior party.

On the estoppel issue, Broad requested judgment against UC because the earlier interference should have decided all issues between the parties. Broad argued that UC should be barred from pursuing its new claims, because Broad's claims involved in the two proceedings are the same, and the same issues based on the same facts were already litigated in the first interference. The PTAB denied Broad's request, explaining that the first interference did not involve priority or patentability of either party, and did not decide those issues, much less all issues that may be argued before the Board. The PTAB was also not persuaded that the counts in the two interferences were directed to the same subject matter. In reaching its decision, the PTAB had to distinguish several USPTO authorities, which might provide Broad with procedural arguments on appeal if it loses priority.

On the third issue, Broad moved for the PTAB to interpret its claims broadly and to broaden the current count defining the scope of disputed subject matter and the proofs for priority. Any claim involved in the interference that is not patentably distinct from the count is grouped with the count. As a result, the losing party would lose any claims patentably indistinct from the count. In the current interference, the original count was directed to a eukaryotic cell comprising CRISPR-Cas9 system with a single guide RNA. Broad requested PTAB to construe its claims reciting "guide RNA" as encompassing both single guide RNA and dual guide RNA, and to substitute the original count with one that covers both single and dual guide RNAs. Broad presumably requested this change because its earliest proofs of invention involved the use of dual guide RNA. The PTAB considered the parties' arguments and declarations and denied these motions, mainly based on Broad's own applications, which stated that "chimeric RNA," "chimeric guide RNA," "single guide RNA,"all of which mean a single guideand the term "guide RNA" are used interchangeably.

Broad moved alternatively for numerous claimsclaims that do not recite a single guide, claims that recite a Cas9 derived from a different species than that was in the provisional applications, and claims that recite two nuclear localization sequencesto be deemed outside of the scope of the original count. The PTAB held that Broad's arguments that these claims were patentably distinct from the count were not supported.

Both UC and Broad have remarked on the PTAB's decision. A UC spokesperson noted that the university, while disagreeing with the accordance of benefits, is pleased that the PTAB has ruled in its favor in most instances, and "remains confident that the PTAB will ultimately recognize that [UC and its affiliated research team] was first to invent the CRISPR-Cas9 technology in eukaryotic cells."2Broad, on the other hand, largely called for a settlement for the parties to move beyond the disputes and "focus on using CRISPR technology to solve today's real-world problems."3

While the PTAB decision declared Broad as the senior party, Broad's priority date is only 47 days before UC's. The interference will now move to the priority phase, where each party may present evidencefor example, actual lab recordsto establish its actual date of the invention. The PTAB has provided time periods for submission of priority motions and oppositions to the motions. These time periods go until May 7, 2021, after which an oral hearing may be ordered. Although Broad won the status of senior party, the retention of the (narrower) original count might impair Broad's ability to contest priority, and certainly its inability to convince the PTAB to end the interference without a priority contest appears to be a significant setback for Broad. This mix of outcomeswith Broad receiving an advantage on priority but with UC prevailing on the terms of the priority contestleaves both parties with considerable uncertainty, possibly increasing pressure to settle.

[1] Patent Trial and Appeal Board Hears Argument in CRISPR Patent Priority Dispute, Wilson Sonsini Alert, https://www.wsgr.com/en/insights/patent-trial-and-appeal-board-hears-argument-in-crispr-patent-priority-dispute.html.

[2] See Jon Cohen, The Latest Round In The CRISPR Patent Battle Has an Apparent Victor, but The Fight Continues, Science (September 11, 2020), https://www.sciencemag.org/news/2020/09/latest-round-crispr-patent-battle-has-apparent-victor-fight-continues.

[3] For Journalists: Statements and Background on the Crispr Patent Process, Broad Communications, https://www.broadinstitute.org/crispr/journalists-statement-and-background-crispr-patent-process (last updated September 10, 2020).

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Patent Trial and Appeal Board Issues Decision on CRISPR Patent Priority Dispute - JD Supra

The research study presented in this report offers complete and intelligent analysis of the competition, segmentation, dynamics, and geographical advancement of the Global Japan CRISPR/Cas9 Market. The research study has been prepared with the use of in-depth qualitative and quantitative analyses of the global Japan CRISPR/Cas9 market. We have also provided absolute dollar opportunity and other types of market analysis on the global Japan CRISPR/Cas9 market.

It takes into account the CAGR, value, volume, revenue, production, consumption, sales, manufacturing cost, prices, and other key factors related to the global Japan CRISPR/Cas9 market. All findings and data on the global Japan CRISPR/Cas9 market provided in the report are calculated, gathered, and verified using advanced and reliable primary and secondary research sources. The regional analysis offered in the report will help you to identify key opportunities of the global Japan CRISPR/Cas9 market available in different regions and countries.

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The authors of the report have segmented the global Japan CRISPR/Cas9 market as per product, application, and region. Segments of the global Japan CRISPR/Cas9 market are analyzed on the basis of market share, production, consumption, revenue, CAGR, market size, and more factors. The analysts have profiled leading players of the global Japan CRISPR/Cas9 market, keeping in view their recent developments, market share, sales, revenue, areas covered, product portfolios, and other aspects.

segment by Type, the product can be split intoGenome EditingGenetic engineeringgRNA Database/Gene LibrarCRISPR PlasmidHuman Stem CellsGenetically Modified Organisms/CropsCell Line Engineering

Market segment by Application, split intoBiotechnology CompaniesPharmaceutical CompaniesAcademic InstitutesResearch and Development Institutes

Based on regional and country-level analysis, the CRISPR/Cas9 market has been segmented as follows:North AmericaUnited StatesCanadaEuropeGermanyFranceU.K.ItalyRussiaNordicRest of EuropeAsia-PacificChinaJapanSouth KoreaSoutheast AsiaIndiaAustraliaRest of Asia-PacificLatin AmericaMexicoBrazilMiddle East & AfricaTurkeySaudi ArabiaUAERest of Middle East & Africa

In the competitive analysis section of the report, leading as well as prominent players of the global CRISPR/Cas9 market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.The key players covered in this studyCaribou BiosciencesIntegrated DNA Technologies (IDT)CRISPR TherapeuticsMerckMirus BioEditas MedicineTakara BioThermo Fisher ScientificHorizon Discovery GroupIntellia TherapeuticsAgilent TechnologiesCellectaGenScriptGeneCopoeiaSynthego

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Japan CRISPR/Cas9 Market Size and Forecast

In terms of region, this research report covers almost all the major regions across the globe such as North America, Europe, South America, the Middle East, and Africa and the Asia Pacific. Europe and North America regions are anticipated to show an upward growth in the years to come. While Japan CRISPR/Cas9 Market in Asia Pacific regions is likely to show remarkable growth during the forecasted period. Cutting edge technology and innovations are the most important traits of the North America region and thats the reason most of the time the US dominates the global markets. Japan CRISPR/Cas9 Market in South, America region is also expected to grow in near future.

The Japan CRISPR/Cas9 Market report highlights is as follows:

This Japan CRISPR/Cas9 market report provides complete market overview which offers the competitive market scenario among major players of the industry, proper understanding of the growth opportunities, and advanced business strategies used by the market in the current and forecast period.

This Japan CRISPR/Cas9 Market report will help a business or an individual to take appropriate business decision and sound actions to be taken after understanding the growth restraining factors, market risks, market situation, market estimation of the competitors.

The expected Japan CRISPR/Cas9 Market growth and development status can be understood in a better way through this five-year forecast information presented in this report

This Japan CRISPR/Cas9 Market research report aids as a broad guideline which provides in-depth insights and detailed analysis of several trade verticals.

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Japan CRISPR/Cas9 Market size and Key Trends in terms of volume and value 2019-2026 - The Daily Chronicle

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Sept. 22, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated(Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).

PRIME is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize development plans and speed up evaluations so these medicines can reach patients faster. The goal of PRIME is to help patients benefit as early as possible from innovative new therapies that have demonstrated the potential to significantly address an unmet medical need. PRIME designation was granted based on clinical data from CRISPR and Vertexs ongoing Phase 1/2 trial of CTX001 in patients with severe SCD.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from transfusion-dependent beta thalassemia (TDT) or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug Designation from the European Commission, for both TDT and SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. CTX001 is the most advanced gene-editing approach in development for TDT and SCD.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) and discussions with regulatory authorities related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the benefits of PRIME designation; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading Risk Factors in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding CTX001s PRIME designation or its development, the potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR Therapeutics and Vertex Pharmaceuticals Announce Priority Medicines (PRIME) Designation Granted by the European Medicines Agency (EMA) to...